RT Journal
A1 Friedrich MJ
T1 SEeing is believing
JF JAMA
JO JAMA
YR 2010
FD October 13
VO 304
IS 14
SP 1543
OP 1545
DO 10.1001/jama.2010.1412
UL http://dx.doi.org/10.1001/jama.2010.1412
AB
Studies of the use of gene therapy to correct defective genes responsible for disease found the approach to be safe and effective in animal models as well as in a limited number of clinical studies. But while genetically engineered viral vectors are adept at penetrating cells and efficiently transferring genes, they also have the potential to elicit negative effects, such as damage to immune responses, that can cause illness or death. Retinal gene therapy trials have relied on viral vectors, and so far aggressive challenges from the immune system have not been reported, perhaps due to the immune-privileged status of the eye. Nevertheless researchers continue to search for better and safer gene delivery vectors, including nonviral vectors such as peptides with cell-penetrating properties, to deliver genes in preclinical ocular gene therapy studies.